Directed Gene Modification via Triple Helix Formation

Title: Directed Gene Modification via Triple Helix Formation

Volume: 1 Issue: 3

Author(s): Linda Gorman and Peter M. Glazer

Affiliation:

Keywords: triplex forming, oligonucleotides (TFOs), peptide nucleic acids (PNAs), Anti-Gene Strategy, TFO-Targeted Mutagenesis, Peptide Nucleic Acids

Abstract: The ability to selectively target mammalian genes and disrupt or restore their function would represent an important advance in gene therapy. Mutation of a single nucleotide can often result in a non-functional gene product. Reversion of defective genes to their correct sequences could lead to permanent cures for patients with many genetic diseases. Molecules such as triplex forming oligonucleotides (TFOs) and peptide nucleic acids (PNAs) are currently being employed to bind to double-stranded DNA. Efficient targeting of genomic DNA with these molecules will be the initial step in gene modification.

Cite this article as:

Gorman Linda and Glazer M. Peter, Directed Gene Modification via Triple Helix Formation, Current Molecular Medicine 2001; 1 (3) . https://dx.doi.org/10.2174/1566524013363771